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Trial record 2 of 3 for:    lysogene | Sanfilippo Syndrome

Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy

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ClinicalTrials.gov Identifier: NCT02053064
Recruitment Status : Completed
First Posted : February 3, 2014
Last Update Posted : June 20, 2017
Sponsor:
Information provided by (Responsible Party):
LYSOGENE

Tracking Information
First Submitted Date  ICMJE January 16, 2014
First Posted Date  ICMJE February 3, 2014
Last Update Posted Date June 20, 2017
Study Start Date  ICMJE May 2013
Actual Primary Completion Date May 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 30, 2015)
Checking of adverse events [ Time Frame: until 60 months after SAF-301 administration ]
Original Primary Outcome Measures  ICMJE
 (submitted: January 31, 2014)
Checking of adverse events [ Time Frame: until 32 months after SAF-301 administration ]
Change History Complete list of historical versions of study NCT02053064 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 30, 2015)
  • Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments [ Time Frame: 5 years after the injection of SAF-301 ]
  • Information on changes in potential biomarkers of the disease [ Time Frame: 5 years after the injection of SAF-301 ]
  • Information to further evaluation of immune response [ Time Frame: 5 years after the injection of SAF-301 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: January 31, 2014)
  • Information on neurological and cognitive changes based on clinical status, standardized neurocognitive and behavioral assessments [ Time Frame: 2 years after the injection of SAF-301 ]
  • Information on changes in potential biomarkers of the disease [ Time Frame: 2 years after the injection of SAF-301 ]
  • Information to further evaluation of immune response [ Time Frame: 2 years after the injection of SAF-301 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
Official Title  ICMJE Long-term Follow-up of Patients With Sanfilippo Type A Syndrome Who Have Previously Been Treated in the P1-SAF-301 Clinical Study Evaluating the Tolerability and Safety of the Intracerebral Administration of SAF-301.
Brief Summary

P2-SAF-301 is an open-label interventional study without administration of investigational product, evaluating the long-term safety and tolerability of intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome.

The primary objective is to collect additional safety and tolerability data on intracerebral SAF-301 previously administered to 4 patients with Sanfilippo type A syndrome.

The secondary objective is to further collect data to assess the effects of SAF-301 on neurological and psychological status, and potential biological markers.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Mucopolysaccharidosis Type III A
  • Sanfilippo Disease Type A
Intervention  ICMJE Genetic: SAF-301
Study Arms  ICMJE Experimental: SAF-301
Intervention: Genetic: SAF-301
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 31, 2014)
4
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE June 2017
Actual Primary Completion Date May 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Patients having completed the previous phase I/II study (P1-SAF-301),
  • Family understanding the follow-up procedures and the informed consent,
  • Patient's parents having signed the informed consent form.

Exclusion Criteria:

  • Participation in any other clinical trial.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02053064
Other Study ID Numbers  ICMJE P2-SAF-301
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party LYSOGENE
Study Sponsor  ICMJE LYSOGENE
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Kumaran DEIVA, Dr AP-HP Hôpital Bicêtre
PRS Account LYSOGENE
Verification Date June 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP